infantile Refsum disease

MeSH: D052919ORPHA: 772

Overview

medical condition

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with infantile Refsum disease, sourced from HPO and Orphanet clinical annotations.

Visual impairmentRod-cone dystrophyNyctalopiaConstriction of peripheral visual fieldGlobal developmental delayFailure to thriveHepatomegalyProgressive muscle weaknessShort statureVery long chain fatty acid accumulationElevated circulating phytanic acid concentrationHearing impairmentSensorineural hearing impairmentNystagmusAtypical behaviorAtaxiaHypotoniaSpasticityAbnormality of the faceCataractOptic atrophySeizureCardiomyopathyAbnormal epiphysis morphologyIchthyosisFacial palsyArrhythmia

Classification & Codes

MeSH Code

D052919

Orphanet Code

ORPHA:772
infantile Refsum disease
MeSHD052919
OrphanetORPHA:772
Treatments0 drug(s)
Symptoms on record27 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
infantile Refsum disease | OrphanDrug